TOPOTECAN for Retinoblastoma

Inclusion criteria

• {"criterion_text":"- Overall study inclusion criteria: Newly diagnosed RB."}
• {"criterion_text":"- Study 2 inclusion criteria: RB eligible for conservative management although not manageable with IAC: a. Unilateral RB in children below 6 months of age, classified as Group A, B, C or D, with or without vitreous seeding, compatible with conservative management, or b. Bilateral RB classified as group A, B, C D, or E without invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye and eligible for conservative management."}
• {"criterion_text":"- Overall study inclusion criteria: RB with at least one eye eligible for conservative management"}
• {"criterion_text":"- Overall study inclusion criteria: Patients likely to be compliant with the study requirements and visits, including late follow-up."}
• {"criterion_text":"- Overall study inclusion criteria: Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer."}
• {"criterion_text":"- Overall study inclusion criteria: Patients with no contraindication to the proposed treatments."}
• {"criterion_text":"- Overall study inclusion criteria: Informed consent signed by parents or legal representative"}
• {"criterion_text":"- Overall study inclusion criteria: French Social Security System coverage."}
• {"criterion_text":"- Study 1 inclusion criteria:Children aged from 6 months to 6 years."}
• {"criterion_text":"- Study 1 inclusion criteria: RB in at least one eye, deemed manageable with IAC in one side and without IV chemotherapy: a. Unilateral RB classified as group B, C (if vitreous seeds < 3 mm), D or E with no invasion of the anterior segment, and without massive tumor of more than 2/3 of the eye, eligible for conservative management, or b. Bilateral RB but with very unbalanced lesions between the two eyes: one eye group D or E without invasion of the anterior segment or massive tumor of more than 2/3 of the eye, eligible for IAC, and the other eye eligible for local treatment only (without IAC)."}

Exclusion criteria

• {"criterion_text":"- Overall study non-inclusion criteria: RB not eligible for conservative management : a. Extra-ocular extension of the disease, or b. Group E eyes with invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye."}
• {"criterion_text":"- Study 1 non-inclusion criteria: Any contraindication or concomitant disease that would preclude the Study 1 treatment procedure and could delay treatment."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patient older than 6 years of age."}
• {"criterion_text":"- Overall study non-inclusion criteria:Patients with another associated disease contra indicating systemic chemotherapy."}
• {"criterion_text":"- Overall study non-inclusion criteria: Previously treated retinoblastoma by chemotherapy"}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients already treated for another malignant disease"}
• {"criterion_text":"- Overall study non-inclusion criteria: Patient with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule."}
• {"criterion_text":"- Overall study non-inclusion criteria: Patients whose parents have not accepted the treatment regimen after explanation of it."}
• {"criterion_text":"- Overall study non-inclusion criteria: Contraindication to study drug mentioned in SmPC (Summary of Products Characteristics) of the study drugs"}
• {"criterion_text":"- Overall study non-inclusion criteria: Inclusion in another experimental anti-cancer drug therapy"}

Primary endpoints

• {"endpoint_text":"- Study 1: Rate of eye preservation in the investigational arm (Topotecan + Melphalan) and in the reference arm (Melphalan) 24 months after the date of randomization.","definition_or_measurement_approach":"Rate of eye preservation assessed 24 months after randomization; comparison between investigational arm (Topotecan + Melphalan) and reference arm (Melphalan)."}
• {"endpoint_text":"- Study 2: Percentage of patients with major, mild or no impairment of visual function according to WHO criteria, i.e. normal bilateral visual acuity (≥ 6/10) or mild bilateral visual impairment (3/10 to 5/10) according to tumor location and extension, when the patient will have 6 years of age and at least 24 months of follow-up after study inclusion.","definition_or_measurement_approach":"Percentage of patients classified by WHO visual function criteria (normal ≥6/10, mild 3/10–5/10, major impairment as per WHO) evaluated at age 6 years and with at least 24 months follow-up after inclusion."}

France

Earliest CTIS Part Ii Submission Date

03-05-2024

Latest Decision Or Authorization Date

31-05-2024

Processing Time Days

28

Number Of Sites

28

Number Of Participants

225

Primary sponsor

Full Name

Institut Curie

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France